WHAT IS batten disease?

Batten Disease is a rare neurodegenerative disease causing seizures, vision loss, cognitive impairment and early death. There is no cure... yet. Parents are forced to make excruciatingly painful decisions as the disease takes its course. Eventually, these families are left to face life without their child.

How common is batten diseasE?

Every year, hundreds of children are born with some variant of Batten Disease, all of which are fatal and have no cure. Worldwide, roughly 14,000 children are afflicted with this disease. Siblings also have a 25% chance of developing the disease, which makes the diagnosis all the more devastating for families as they wonder the fate of their other children.

the science behind batten

In Batten Disease, a simple gene mutation prevents cells from breaking down fats and proteins, leading to a build-up in the cells' trashcans, or lysosomes. This results in cell death and the body’s inability to function normally. The condition is autosomal recessive, meaning both parents must carry the same abnormal gene in order for their children to develop the disease. There are fourteen known variants of Batten - CLN1, CLN2, and so on - and each is unique enough to require its own research and trials. We are focused on Mila’s variant, CLN7.

To learn more about Batten Disease, visit:

IS THERE A TREATMENT?

There is no cure for Batten Disease... yet. However, we are working hard to start and fund new treatments that could help children with Batten and other devastating neurological disorders. Our work is for Mila and for the millions of other children like her that deserve a chance. 

Through our efforts at Mila’s Miracle Foundation, there is now a CLN7 Gene Replacement Therapy in trial at UT Southwestern Medical Center. This clinical trial was made possible based off the work that we started, with help from Batten Hope foundation and Dr. Steven Gray.

As a result of the work we initiated toward a novel gene therapy, there is now a CLN7 Natural History Study underway as well. The purpose of this study is to collect data from children to better understand the disease and help advance treatments.

For more information about the CLN7 Natural History Study, please contact Neurogene:

To learn more about active gene therapies and clinical studies for ALL variants of Batten disease, visit clinicaltrials.gov.